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Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma

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ClinicalTrials.gov Identifier: NCT02017964
Recruitment Status : Active, not recruiting
First Posted : December 23, 2013
Results First Posted : April 4, 2019
Last Update Posted : June 5, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Desmoplastic/Nodular Medulloblastoma
Medulloblastoma
Medulloblastoma With Extensive Nodularity
Nevoid Basal Cell Carcinoma Syndrome
Untreated Childhood Medulloblastoma
Interventions Drug: Carboplatin
Other: Cognitive Assessment
Drug: Cyclophosphamide
Drug: Etoposide
Other: Laboratory Biomarker Analysis
Drug: Methotrexate
Drug: Vincristine Sulfate
Enrollment 26
Recruitment Details This study opened for entry on December 24, 2013, and the first patient was enrolled on April 22, 2014. The study enrolled 26 patients before being closed early due to inferior efficacy as of July 27, 2016. 25 of the 26 patients enrolled were deemed to be eligible
Pre-assignment Details  
Arm/Group Title All Patients (Combination Chemotherapy)
Hide Arm/Group Description All patients enrolled on the study. Patients received 3-5 cycles of therapy which included cyclophosphamide (800 mg/m2/day), vincristine (1.5 mg/m2/day), methotrexate (5,000 mg/m2), etoposide (150 mg/m2/dose) and carboplatin (200 mg/m2/day IV).
Period Title: Overall Study
Started 26
Completed 25
Not Completed 1
Reason Not Completed
Patient deemed ineligible             1
Arm/Group Title All Eligible Patients (Combination Chemotherapy)
Hide Arm/Group Description All eligible patients enrolled on the study
Overall Number of Baseline Participants 26
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants
<=18 years
26
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Months (at time of dx)
Number Analyzed 26 participants
19.7
(7.1 to 42.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants
Female
10
  38.5%
Male
16
  61.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants
Hispanic or Latino
6
  23.1%
Not Hispanic or Latino
19
  73.1%
Unknown or Not Reported
1
   3.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   3.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
   7.7%
White
20
  76.9%
More than one race
0
   0.0%
Unknown or Not Reported
3
  11.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 26 participants
United States 23
Canada 3
1.Primary Outcome
Title Progression-free Survival (PFS)
Hide Description Progression-free survival (PFS) is defined as the time interval from diagnosis to the earliest of disease progression/recurrence or death from any cause, or to the date of last follow-up for patients without events. PFS was estimated using the method of Kaplan and Meier. 2-year estimates are reported with 95% CI's.
Time Frame 2 years from diagnosis
Hide Outcome Measure Data
Hide Analysis Population Description
26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient was excluded from all analyses.
Arm/Group Title All Eligible Patients (Combination Chemotherapy)
Hide Arm/Group Description:
All eligible patients enrolled on the study
Overall Number of Participants Analyzed 25
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percent probability
52.0
(32.4 to 71.6)
2.Primary Outcome
Title Overall Survival (OS)
Hide Description Overall Survival (OS) is defined as the time from diagnosis to death from any cause, or to the date of last follow-up for survivors. OS was estimated using the method of Kaplan and Meier. 2-year estimates are reported with 95% CI's, as the data are not mature to 72 months.
Time Frame Assessed up to 72 months, reported at 2 years from diagnosis
Hide Outcome Measure Data
Hide Analysis Population Description
26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient was excluded from all analyses.
Arm/Group Title All Eligible Patients (Combination Chemotherapy)
Hide Arm/Group Description:
All eligible patients enrolled on the study
Overall Number of Participants Analyzed 25
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percent probability
92.0
(80.8 to 100.0)
3.Primary Outcome
Title Event-free Survival (EFS)
Hide Description Event-free survival (EFS) is defined as the time from diagnosis to the earliest of disease progression/recurrence, second malignancy or death from any cause, or to the date of last follow-up for patients without events. EFS was estimated using the method of Kaplan and Meier. 2-year estimates are reported with 95% CI's.
Time Frame 2 years from diagnosis
Hide Outcome Measure Data
Hide Analysis Population Description
26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient is excluded from all analyses.
Arm/Group Title All Eligible Patients (Combination Chemotherapy)
Hide Arm/Group Description:
All eligible patients enrolled on the study
Overall Number of Participants Analyzed 25
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percent probability
52.0
(32.4 to 71.6)
4.Primary Outcome
Title Percentage of Patients With Responses at 189 Days
Hide Description The percentage of patients with complete response (CR) at the end of induction (~189 days) was reported and presented with the associated exact 95% confidence interval.
Time Frame 189 days from start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient was excluded from all analyses.
Arm/Group Title All Eligible Patients (Combination Chemotherapy)
Hide Arm/Group Description:
All eligible patients enrolled on the study
Overall Number of Participants Analyzed 25
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of patients
92.0
(75.6 to 98.6)
5.Primary Outcome
Title Percentage of Patients With Responses at 273 Days
Hide Description The percentage of patients with complete response (CR) at the end of therapy (~273 days) was reported and presented with the associated exact 95% confidence interval.
Time Frame 273 days from start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
26 patients were enrolled on the protocol, but 1 was deemed ineligible. The ineligible patient is excluded from all analyses.
Arm/Group Title All Eligible Patients (Combination Chemotherapy)
Hide Arm/Group Description:
All eligible patients enrolled on the study
Overall Number of Participants Analyzed 25
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of patients
88.0
(69.7 to 96.7)
6.Other Pre-specified Outcome
Title Feasibility of Rapid Central Pathology Screening Review Defined as Success Rate of Timely Central Pathology Review Based on the Expectation That at Least 95% of the Cases Will be Reviewed Within 10 Days
Hide Description At the end of the trial the feasibility of such a prescreening process will be assessed by reporting the percentage and the associated confidence interval of cases where the central review was obtained within 10 days from receipt of slides.
Time Frame Up to 10 days
Outcome Measure Data Not Reported
7.Other Pre-specified Outcome
Title Molecular Profile
Hide Description Contingency table analysis will be used to examine the association of molecular profile with histology.
Time Frame Up to 2 years
Outcome Measure Data Not Reported
8.Other Pre-specified Outcome
Title Change in Neurocognitive and Adaptive Functioning Assessed Using Full Scale IQ Score (FSIQ) and General Adaptive Composite Score (GAC)
Hide Description Changes will be described via paired tests and confidence intervals both for FSIQ and GAC in order to capture any deterioration over time.
Time Frame Baseline to up to 60 months
Outcome Measure Data Not Reported
Time Frame 1 year
Adverse Event Reporting Description Adverse event reporting is collected routinely using case report forms. SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Ineligible patients are excluded from reporting of adverse events.
 
Arm/Group Title All Eligible Patients (Combination Chemotherapy)
Hide Arm/Group Description All eligible patients enrolled on the study
All-Cause Mortality
All Eligible Patients (Combination Chemotherapy)
Affected / at Risk (%)
Total   2/25 (8.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
All Eligible Patients (Combination Chemotherapy)
Affected / at Risk (%) # Events
Total   2/25 (8.00%)    
General disorders   
Death NOS   1/25 (4.00%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Other, Progression Of Medulloblastoma   1/25 (4.00%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
All Eligible Patients (Combination Chemotherapy)
Affected / at Risk (%) # Events
Total   17/25 (68.00%)    
Blood and lymphatic system disorders   
Febrile neutropenia   1/25 (4.00%)  1
Leukocytosis   1/25 (4.00%)  4
Ear and labyrinth disorders   
Hearing impaired   1/25 (4.00%)  1
Infections and infestations   
Meningitis   1/25 (4.00%)  1
Investigations   
Lymphocyte count decreased   7/25 (28.00%)  18
Neutrophil count decreased   14/25 (56.00%)  36
Platelet count decreased   7/25 (28.00%)  13
White blood cell decreased   11/25 (44.00%)  24
Metabolism and nutrition disorders   
Hypernatremia   1/25 (4.00%)  1
Hypokalemia   1/25 (4.00%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Must obtain prior Sponsor approval
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Results Reporting Coordinator
Organization: Children's Oncology Group
Phone: 626-446-0064
EMail: resultsreportingcoordinator@childrensoncologygroup.org
Layout table for additonal information
Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT02017964     History of Changes
Other Study ID Numbers: ACNS1221
NCI-2013-02379 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
ACNS1221
COG-ACNS1221
s15-00476
ACNS1221 ( Other Identifier: Childrens Oncology Group )
ACNS1221 ( Other Identifier: CTEP )
U10CA180886 ( U.S. NIH Grant/Contract )
U10CA098543 ( U.S. NIH Grant/Contract )
First Submitted: December 13, 2013
First Posted: December 23, 2013
Results First Submitted: March 12, 2019
Results First Posted: April 4, 2019
Last Update Posted: June 5, 2019